Stem Cells— i on NIH — episode #0015, segment 1

Video of Stem Cells -- i on NIH -- episode #0015, segment 1

Dr. Story Landis is Director of the National Institute of Neurological Disorders and Stoke, and the chair of the NIH Stem Cell Task Force. She reminds that some cell-based therapies, like bone marrow transplants, are already being used.

Transcript

Welcome to “i on NIH”!

Featured in this month’s episode are segments about stem cells, the opening of the NIH Gateway Building, and an eye-to-eye interview with Dr. Anthony Fauci, the director of the National Institute of Allergy and Infectious Diseases who was recently awarded the Presidential Medal of Freedom.

From the national institutes of health in Bethesda, Maryland – America's premiere medical research agency – this is “i on NIH”!

Covering health-research topics important to you and the nation, this public service vodcast is your information source from inside all 27 institutes and centers at NIH.

Half an hour, once a month, we'll show you the excitement of advances and the important information that comes from medical research.

And now, here's your host, Joe Balintfy.

Host: Welcome to the 15th edition of i on NIH! Thanks for joining us. As you heard from the intro, we have two reports and an eye-to-eye interview in this program: a story about stem cells, a feature on the newest building here at NIH; and an interview about how an institute director received the Presidential Medal of Freedom. But before we get into those, we have this news update from Harrison Wein at the nih news-desk. Harrison…

Chronic kidney disease affects 26 million Americans. It can be caused by many different diseases and conditions. African Americans have a higher risk for several forms of chronic kidney disease, with almost a 4-fold higher risk of one called FSGS. NIH researchers scanned the genomes of 190 African Americans known to have FSGS and compared them to 222 without it. They identified several variations in a gene called MYH9 that add to the higher risk of kidney disease among African Americans. The researchers shared their discovery with scientists at Johns Hopkins who are part of a large consortium doing genetic studies of kidney disease. The Hopkins team studied more than 2,100 people and found that the gene variants are much more frequent among people of African ancestry than whites. 60% of African Americans carry these gene variants, compared to 4% of whites. This finding represents a major step in understanding an important health disparity. It could lead to the development of diagnostic tools to identify people at higher risk for FSGS, and eventually to new therapies to prevent kidney disease from progressing.

Hair cells in our inner ears are a vital part of our hearing. Their loss is a leading cause of hearing impairment. These cells turn the vibrations of sound into electrical signals that travel to the brain. Once they're damaged, the body can't replace them. Over the past few years, researchers have made significant progress in understanding how these cells work. Researchers at the Oregon Health and Science University have now used gene transfer to produce functional hair cells in the ears of mice. They devised a method for inserting genes into the region of embryonic mice that develops into the inner ear. Then they transferred a gene that's needed for hair-cell formation into the region. The genetically altered mice had a nearly 2-fold increase in inner ear hair cells. The cells appeared to be fully functional. The next step will be to use the technique to try to treat hearing loss in mouse models of human deafness and balance disorders. Ultimately, this research may lead to therapies for treating hearing problems in people.

A large-scale, comprehensive study of glioblastoma, the most common form of brain cancer, has uncovered new DNA alterations involved with the disease. Until recently, scientists have understood little about what causes glioblastoma. In the new study, 10 different academic centers and over 125 investigators worked together to analyze the genomes, or complete sets of DNA, of tumor samples donated by over 200 patients with glioblastoma. They identified many gene mutations involved in the cancer, including 3 frequent but previously unrecognized mutations. They also found 3 core biological pathways that likely play a role in glioblastoma. Each pathway was disrupted in more than three-quarters of the tumors. The more we learn about the molecular basis of glioblastoma, the sooner we can develop better ways of helping patients with the disease. To hear more about this study, check out the September 19th podcast of NIH's "Research Radio." And to read more about these and many other research studies in "NIH Research Matters." Go to the NIH home page and look for the link on the right-hand side, under "In the News" that says, "eColumn: NIH Research Matters" And in this month's health newsletter, "NIH News in Health"… Helping your kids eat smart at school, planning for the school year if your child has diabetes, and more in the September issue of NIH News in Health. You can find it at news-in-health-dot-nih-gov. This is Harrison Wein at the NIH Science Desk. Back to you, Joe.

Host: Thanks Harrison. Again, be sure to check out the podcast episode from September 19 that Harrison mentioned. It includes an in-depth interview about the Cancer Genome Atlas Report. Now for our first feature in this vodcast, we learn about stem cells and get an inside perspective on what research is showing.

VO: At an NIH symposium in May, researchers shared examples of state-of-the-art stem cell-based therapies. Stem cells have been touted as a toolkit to treat a variety of diseases.

Landis: It’s very promising…

VO: Dr. Story Landis is Director of the National Institute of Neurological Disorders and Stoke, and the chair of the NIH Stem Cell Task Force. She reminds that some cell-based therapies, like bone marrow transplants, are already being used.

Landis: …there are some cell-based therapies, particularly for blood diseases, which are FDA approved and in wide practice and represent a model for how we hope other cell-based therapies will develop. But, for example, for nervous system diseases, we are at a very early stage and a lot of research needs to be done before we have cell-based therapies.

VO: The Symposium, titled Challenges and Promise of Cell-Based Therapies, was attended by roughly 400 scientists who heard presentations and discussed clinical applications of embryonic and adult stem cells. The goal being to determine productive new directions for research in regenerative medicine and to help NIH prioritize research with the greatest potential for clinical benefit. Dr. Landis mentions, as an example, the speakers who discussed nervous system applications.

Landis: I thought they did an excellent job of showing, on the one hand, that, for multiple sclerosis and autoimmune disease, it’s an interesting possible application of hematopoietic stem cells to replace the immune cells that are attacking myelin. But when you get into nuero-degenerative diseases like Parkinson’s and amyotrophic lateral sclerosis, that kind of strategy is unlikely to work, and we’ll need to think much more broadly about replacing nervous system cells with stem cells from the nervous system.

VO: Not all stem cells are the same. Embryonic stem cells are pluripotent, meaning they can develop into any cell type. Stem cells are also found in adult tissues, but whether or not they are truly pluripotent is not known. Lesley Stewart, Science Policy Advisor and Director of Legislative Affairs at the National Institute on Deafness and Other Communication Disorders, explains the different types of stem cells.

Stewart: So, an induced pluripotent stem cell, the term that they gave it, is a result of reprogramming, and what happens during that process is they take, say, a skin cell and they use certain factors to revert it back to an embryonic-like state. And they use sort of a chemical mix to do that, and by doing so it’s similar to an embryonic stem cell, but more research needs to be done to decide what the differences are between those two types of stem cells. Whereas embryonic stem cells have the most potential in terms of differentiation, they can differentiate into all the different types of cell types, whereas adult stem cells are somewhat limited in the type of cell type they can differentiate into. And then there was also mention of cord blood stem cells and amniotic stem cells. Each has a different area of origin, and each has a different ability to differentiate it to a different cell type. So that’s sort of a breakdown of the different types of stem cells.

VO: Experts agree there is no single "best" stem cell for all applications; rather, a wide range of options is available. Stewart adds that more research must be done to learn how the different types of stems cells work before treatments will be available.

Stewart: Right, we’re many years off from therapeutic applications. In terms of other types of information that we can learn from stem cells, I would say that drug toxicity testing, which is being used currently, its helpful if you use, say -- if you differentiate a stem cell to a cardiomyocite, and then you’re able to test a certain drug on a human cell instead of testing an animal model. That can currently be done, and that would be very beneficial and more efficient in terms of deciding how is that going to impact a potential patient. So, there are areas that can be used currently, like I said with adult stem cells, bone marrow transplants have been done for years. So, there are areas right now that are being used. There still is a lot of work that needs to be done, and there is a need to research all types of stem cells to decide which has the best potential for therapeutic application because at this point we don’t know which type of stem cell is going to have the best possibility to treat a given disease or disorder.

VO: Researchers are exploring basic science but also looking at translational science and clinical science to learn how the best treatments can be brought to people that are in need.

Stewart: And it’s not going to happen in a year or two years. It might be 10 years away, but there are going to be potential discoveries that’s going to revolutionize medicine as we know it now. So, just in terms of even the knowledge we obtain from learning how a cell decides to differentiate and become the different types of cells that it does, and there’s all types of knowledge that we don’t have that we can potentially learn through more and more research in that area.

VO: Both Dr. Landis and Stewart emphasize the importance of the science.

Landis: My sense from press coverage of anything to do with stem cells, is that it very quickly gets away from the science and into political issues, which actually make it difficult to understand what the real state of the science is.

Stewart: Be cautious about some of the information that you get in the media and to know that there are both sides to the issue, that some are very, very positive and some are very, very negative, and to just take it with caution and be a very conscientious consumer of information. And just to know that, yes, there’s a lot of potential with stem cell therapies. We’re far off from those being readily available in the clinic at this point, but with additional research and with more and more research towards this area, there is a huge potential in the future for this to treat a variety of diseases and disorders.

VO: Stewart also points out that NIH supports all types of research on all types of stem cells under the current policy. She adds that much more information about stem cells is available online.

Stewart: Visit the NIH stem cell Website. We have a comprehensive overview of policy, ethics, recent science advances, all kinds of helpful information, educational resources on our website, we update it frequently, and funding opportunity announcements. So, I would just really encourage people to visit our Website and to learn as much as they can about stem cell research through that means.

VO: The website is stemcells.nih.gov.

Host: You may know that the National Institutes of Health is located in Bethesda, Maryland. The campus is just north of Washington, DC and inside the capital beltway, interstate 495. The summer of 2008 marked the opening of the new NIH Gateway Center on the campus. And for visitors, it will likely be the first building they enter.

VO: Sergeant Bob Drummond is with the NIH Police. He summarizes what the Gateway Center is.

Drummond: It’s a screening and processing area for all visitors coming on to campus whether by foot, metro, bus, walking, and for vehicle traffic.

VO: Chuck Crawford, project architect for the Gateway Center explains how the facility is really three buildings in one.

Crawford: One is a parking facility; the other is a visitor building, which is this building that we’re currently in. And the third facility is a vehicle inspection building.

VO: MLP-11 is the multi-level parking garage outside of campus and mostly underground. The visitor building, building 66, is for pedestrians entering campus. The inspection station, building 66A, is for cars entering campus. Overall, the Gateway Center has nearly 140-thousand square feet of space, 342 parking spaces, and cost 31-million dollars. It also has a “green” roof.

Kim: Not color wise, not necessarily over the time, but what -- it’s a vegetation roof. Basically, it’s vegetation on top of a structure. We call it green roof because it’s a vegetation more than anything.

VO: Brian Kim, an Environmental Engineer with NIH Environmental Protection department explains that there are many advantages to having a green roof.

Kim: The obvious ones are the aesthetic value of it, just to have not all the gray concrete, but actually having some green in the building is good. Also, it can be an energy saver because it works as a temperature buffer for extreme cold and warmer climates. It insulates the building from hot temperature in summertime, and it keeps the building warmer in colder temperature. And it also saves energy that way, too, obviously.

Crawford: And I think it makes sense when you think about NIH being a facility that deals with health and well being that a green roof would be a logical move.

VO: Crawford and Kim point out that green roofs reduce the requirement for draining and minimize storm drain require ments. Crawford emphasizes that it’s just one element that makes the Gateway Center a pleasing place.

Crawford: That’s what architecture is supposed to do in many ways, is to create an interesting place. So I think we’ve achieved that. Hopefully the people that use the facility enjoy it, and the people that visit it will be impressed by it, and it will be a good impression on NIH. So that’s what I hope the net result will be.

VO: Crawford adds that building 66 flows with its surroundings.

Crawford: We’ve scaled the building and worked the building into the landscape very successfully, and I think that’s made a much, a very pleasing facility, pleasing to the eye, and just, you know, obviously from a circulation standpoint, I think the building works very well. In fact, this kind of undulating shape is very reflective of the actual circulation pattern, so it’s sort of, I guess, sort of a telegraphic upward of the circulation pattern. That’s hence this kind of curvilinear building.

VO: Sgt. Drummond anticipates that things will flow smoothly on the inside as well. He explains what it will be like for a visitor coming through the new Gateway Center.

Drummond: Very easy. The first thing is they’ll do is they’ll come through x-ray and magnetometer processing, which will only take a few minutes. After processing and they pass screening, they’ll proceed to the badging area. They’ll present their drivers license or passport, any government issued photo identification, provide that to the clerk. The clerk will scan their information, issue a visitor pass, and then they’re free to go onto campus.

VO: For visitors coming by car to campus, they have the choice of parking in the garage, or if they want to drive onto campus, the process is slightly different. Sgt. Drummond explains.

Drummond: They’ll be coming off Rockville Pike into the Gateway inspection center, or Gateway Vehicle Inspection Center. They’ll pull to the front of the facility, they’ll be asked to get out of the car and take their property with them; they’ll go inside a badging station. While they’re getting their visitor pass, their vehicle will be inspected by a physical inspector from our security company. And then they’ll be free to get back in their car and exit the facility.

VO: The National Institutes of Health, like all Federal Government facilities, has instituted security measures to ensure the safety of NIH employees, patients, and visitors. The Gateway Center and its three components are part of that.

Crawford: Functionally they work together in terms of how people get onto the campus and how they’re received in the campus.

VO: For more information on the Gateway Center, its green roof, and how to access the NIH campus, visit www.nih.gov.

Host: Now for our eye-to-eye interview. Dr. Anthony S. Fauci, director of the National Institute of Allergy and Infectious Diseases was honored by President George W. Bush with the Presidential Medal of Freedom at a White House ceremony this past summer. For this eye-to-eye interview we got Dr. Fauci’s perspective…

VO: What does it mean to you to win the Presidential Medal of Freedom?

Fauci: Well it's an extraordinary experience and an extraordinary honor. It's actually quite humbling when you think of the fact that your life's work has been publicly honored by the President of the United States and a medal that's given to people. When you look at the track record and the history of the people who have gotten the Medal of Freedom, it really is very humbling. It also reminds me that despite all that we've accomplished with HIV, which is the primary reason for my getting the Presidential Medal of Freedom, it also reminds you about how much more we need to do.

VO: What challenges do you feel remain to be met regarding HIV research?

Fauci: Well, I think the biggest challenge, there’s a scientific challenge, and it has to do with developing a vaccine. We've been quite successful in developing a whole menu of drugs that have proven to be very effective in controlling HIV infection in people who are already infected. But we know just from the numbers of the numbers of new infections each year -- in the world there are 2.5 million new infections each year -- and yet from every one person that we put on therapy, two to three people get newly infected. So although there's great successes with therapy, numerically you're losing the game if you continue to have so many additional people getting infected. So prevention looms large as a real major challenge, and the scientific aspect of that is developing a vaccine, and vaccine has been very elusive over the last many years for a number of reasons. Most importantly, that HIV is really quite different from any other virus that we have ever tackled in our attempts to develop a vaccine because the body does not seem to naturally make a good protective immune response against HIV. Even all the great killers, small pox, measles, polio, those diseases that were scourges, the body does still make a good immune response against them, and the majority of people seem to recover. So when you make a vaccine, what you do is you mimic what the body's natural response is. Unfortunately with HIV, the body's natural response is terribly inadequate. So that's a big scientific challenge. The big public health challenge is access to the people particularly in the low and middle-income countries to treatment, prevention and care. Because 90% of the infections occur in the low and middle-income countries, and 66 to 68% occur in southern Africa. So we have a public health issue of getting access to those people, and we have a scientific issue, the last of the big scientific issues, is to develop a vaccine.

VO: What was the award ceremony like?

Fauci: Well it was a very personal, warm type of ceremony. It wasn't one of those things that are very official. I mean, I go to the White House a lot to brief everyone from the President to the Vice President to different members of the senior staff, and it’s generally all strictly business. You go in, the people are very nice and they’re very cordial. But this was very personal, personal about the awardees. I was one of six. There were five others who got the awards. And very warm and personal with their family members. We took pictures with the President and the First Lady, and the President took a lot of time to be very cordial with my daughters, with my wife, and chatting a bit and not just trying to get it over with but actually seeming like he was actually enjoying this. This was in many respects his day because this is the highest award that he as President awards. So he seem to be enjoying it as much as the awardees were enjoying it.

VO: Who were some of the other awardees?

Fauci: Yeah, well, Ben Carson, who's a established neurosurgeon at Hopkins and has done ground breaking neurosurgery, particularly in babies who are conjoined at the brain, and he does excellent work on epilepsy. Congressman Tom Lantos’ widow, Tom died recently, and he was one of the big proponents of the President's Emergency Plan for AIDS Relief, which is one of the reasons why I got the award was because of the role that I played in developing that global plan. We had Larry Silverman, one of our judges here in Washington D.C., who is, you know, an extraordinary jurist.

Fauci: We had General Peter Pace, who is former Commandant of the Marine corps and former chairman of the Joint Chiefs of Staff, a Vietnam War veteran, a war hero, and a person who is very much involved in the planning for the war. And we had Donna Shalala, which I found to be very interesting. Donna Shalala is truly a card-carrying, liberal Democrat, and the President was open minded enough and the administration was open minded enough to give her the award. She is the former Secretary of Health and Human Services in the Clinton administration, and she was actually my boss when she was Secretary of HHS. So it was really very interesting that there we were the two of us on the same stage together. When years and years ago during the Clinton administration, she was the secretary of the department that I worked in and that I still work in.

VO: How would you summarize the experience?

Fauci: Well, you know it's just as I mentioned: there’s so many aspects of it that are exciting and wonderful. Probably the one of the ones that is the most gratifying is to be in the company of people who are coming from different disciplines to be sitting there next to General Pace and to be sitting down next to Larry Silverman and others who made major contributions in areas that have nothing to do with what I do is a very good feeling.

VO: For more information on Dr. Fauci’s award and NIAID research, visit www.niaid.nih.gov.

Host: And that’s it for another episode on I on NIH. Thanks for tuning in and please watch again next time. We’re working on stories about global health, brain cells, and much more. For I on NIH, I’m Joe Balintfy.